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Treating hereditary transthyretin amyloidosis: Present & amp; future challenges
Rev Neurol (Paris). 2022 Sep 20:S0035-3787(22)00742-1. doi: 10.1016/j.neurol.2022.07.006. Online ahead of print.ABSTRACTHereditary transthyretin amyloidosis (ATTRv) is a rare, lethal, autosomal dominant adult-onset genetic gain-of function (GOF) disorder provoked by mutations in the TTR gene. Until recently, therapeutic options were limited and consisted mainly in liver transplantation and TTR-stabilizers. In the last few years, ATTRv has been at the center of major therapeutic breakthroughs, including development of effective small interfering RNA (siRNA) and antisense oligonucleotide (ASO) treatments targeting liver TTR ...
Source: Revue Neurologique - September 23, 2022 Category: Neurology Authors: A Echaniz-Laguna C Cauquil C Labeyrie D Adams Source Type: research